Past and current practice of patient-reported outcome measurement in randomized cancer clinical trials: a systematic review

Research output: Contribution to journalArticlepeer-review


  • Johannes M. Giesinger
  • Fabio Efficace
  • Neil Aaronson
  • Francesco Cottone
  • David Cella
  • Eva-Maria Gamper

Colleges, School and Institutes


Objective: In our systematic review, we assessed past and current practice of patient-reported outcome (PRO) measurement in cancer randomized controlled trials (RCTs).

Methods: We included RCTs with PRO endpoints evaluating conventional medical treatments, conducted in patients with the most prevalent solid tumour types (breast, lung, colorectal, prostate, bladder, and gynaecological cancers) and either published 2004-2018 or registered on and initiated 2014-2019. Frequency of use of individual PRO measures was assessed overall, over time, and by cancer site.

Results: Screening of 42,095 database records and 3,425 registered trials identified 480 published and 537 registered trials meeting inclusion criteria. Among published trials, the EORTC measures were used most often (54.8% of trials), followed by the FACIT measures (35.8%), the EQ-5D (10.2%), the SF-36 (7.3%), and the MDASI (2.5%). Among registered trials, the EORTC measures were used in 66.1% of the trials, followed by the FACIT measures (25.9%), the EQ-5D (23.1%), the SF-36 (4.8%), the PRO-CTCAE (2.2%), the PROMIS measures (1.7%), and the MDASI measures (1.1%).

Conclusion: The PRO measures most frequently used in RCTs identified in our review, differ substantially in terms of content and domains, reflecting the ongoing debate among the scientific community, health care providers and regulators on the type of PRO to be measured. Current findings may contribute to better informing the development of an internationally agreed core outcome set for future cancer trials.

Bibliographic note

Conflict of Interest Disclosures: Drs Giesinger and Gamper reported receiving a research grant from the European Organisation for Research and Treatment of Cancer (EORTC) for an unrelated project. Dr Efficace reported receiving personal fees from Abbvie, Bristol Myers Squibb, Orsenix, and Takeda outside the submitted work; grants and personal fees from Amgen outside the submitted work; and a grant from the EORTC Quality of Life Group outside the submitted work. Dr Calvert is a National Institute for Health Research (NIHR) Senior Investigator and reported receiving grants from the NIHR, Health Data Research UK, Macmillan Cancer Support, UCB Pharma, and Innovate UK outside the submitted work; and personal fees from Astellas, Takeda, Merck, GlaxoSmithKline, Daiichi Sankyo, and Glaukos outside the submitted work. Dr Kyte reported receiving grants from NIHR, Innovate UK, NIHR Birmingham Biomedical Research Centre, NIHR Surgical Reconstruction and Microbiology Research Centre at the University of Birmingham, University Hospitals Birmingham National Health Service Foundation Trust, the Macmillan Cancer Support, and Kidney Research UK outside the submitted work; and personal fees from Merck and GlaxoSmithKline outside the submitted work. Dr Cella is the president of No other disclosures were reported. Funding/Support: The authors received no financial support for this research. Acknowledgment: The views expressed in this article are those of the authors and not necessarily those of the NIHR, NHS or the Department of Health and Social Care.


Original languageEnglish
Pages (from-to)1-7
JournalValue in Health
Issue number00
Early online date10 Feb 2021
Publication statusE-pub ahead of print - 10 Feb 2021


  • cancer, patient-reported outcome, questionnaire, randomized-controlled trial, systematic review